ChemoCentryx, Inc., (Nasdaq:CCXI), a biopharmaceutical company
developing new medications targeted at inflammatory and autoimmune
diseases and cancer, announced today that the U.S. Food and Drug
Administration (FDA) has granted orphan drug designation for
avacopan in the treatment of patients with C3 glomerulopathy (C3G).
C3G is a severe kidney disease characterized by deposits of
proteins from the body’s complement system into and around the
kidney’s filtration units, the glomeruli. The deposition of
complement protein disrupts kidney function and also triggers a
profoundly destructive inflammation of the kidney. The disease
process ultimately leads to renal failure, with the need for
dialysis and kidney transplant.
There is currently no approved treatment for C3G, and relapse is
common even after kidney transplant.
Avacopan targets the complement C5a receptor (C5aR) which drives
the destructive inflammatory cells in C3G and other kidney
diseases. In this way, avacopan is designed to arrest the
ongoing process of kidney damage in C3G, allowing kidney function
to stabilize.
“Scientific and clinical evidence supports avacopan’s potential
in the treatment of C3G, a debilitating kidney disorder that
strikes young people,” said Thomas J. Schall, Ph.D., President
and Chief Executive Officer of ChemoCentryx. “Receipt of FDA
orphan status will allow us to expedite development of this
promising treatment option for C3G, which has already had a
life-changing effect on a C3G patient treated continuously with
avacopan for well over a year under the Special Needs program (the
equivalent of a compassionate use protocol) in the United
Kingdom.”
ChemoCentryx plans to initiate a multi-center clinical endpoint
study of avacopan for the treatment of C3G in the first half of
2017.
About Avacopan (CCX168)
Avacopan (CCX168) is an orally-administered small molecule that
is a selective inhibitor of the complement C5a receptor, or
C5aR. Avacopan is in Phase III development for the treatment
of anti-neutrophil cytoplasmic auto-antibody-associated vasculitis
(AAV). In clinical studies to date, avacopan was shown to be safe,
well tolerated and provided effective control of the disease while
successfully allowing elimination of high-dose steroids, part of
the standard of care for AAV patients. Avacopan is also being
developed in patients with C3 glomerulopathy (C3G) and atypical
hemolytic uremic syndrome (aHUS).
The U.S. Food and Drug Administration has now granted
avacopan orphan-drug designation for all three of these diseases:
AAV, C3G and aHUS. The European Commission has granted
orphan medicinal product designation for avacopan for the treatment
of two forms of AAV: microscopic polyangiitis and granulomatosis
with polyangiitis (formerly known as Wegener's granulomatosis).
Avacopan was also granted access to the European Medicines
Agency's (EMA) PRIority MEdicines (PRIME) initiative, which
supports accelerated assessment of investigational therapies
addressing unmet medical need.
About Complement 3 Glomerulopathy (C3G)
C3 glomerulopathy is characterized by evidence of alternative
complement activation based on C3 deposition in the glomeruli.
Patients with C3G often have high levels of protein in the urine
(proteinuria) and experience progressive deterioration in renal
function. Without treatment, C3G leads to kidney failure, with
kidney transplant often the only option. Even after
transplantation, the new kidney will frequently manifest the
disease.
There is no approved treatment for C3G; non-specific
immunosuppressants are frequently employed. The estimated
prevalence of C3G is two-to-three per million people.
About ChemoCentryx
ChemoCentryx is a biopharmaceutical company developing new
medications targeted at inflammatory and autoimmune diseases, and
cancer. ChemoCentryx targets the chemokine and chemoattractant
systems to discover, develop and commercialize orally-administered
therapies. ChemoCentryx is currently focusing on its late stage
drug candidates for patients with rare kidney diseases, avacopan
(CCX168) and CCX140.
Avacopan is an orally-administered small molecule that is a
selective inhibitor of the complement C5a receptor, or C5aR.
Avacopan is in Phase III development for the treatment of
anti-neutrophil cytoplasmic auto-antibody-associated vasculitis
(AAV). In clinical studies to date, avacopan was shown to be safe,
well tolerated and provided effective control of the disease while
allowing elimination of high-dose steroids, part of the current
standard of care. Avacopan is also being developed in patients with
C3 glomerulopathy (C3G) and atypical hemolytic uremic syndrome
(aHUS). The U.S. Food and Drug Administration has now
granted avacopan orphan-drug designation for three indications,
AAV, C3G and aHUS. The European Commission has granted
orphan medicinal product designation for avacopan for the treatment
of two forms of AAV: microscopic polyangiitis and granulomatosis
with polyangiitis (formerly known as Wegener's granulomatosis).
Avacopan was also granted access to the European Medicines
Agency's (EMA) PRIority MEdicines (PRIME) initiative, which
supports accelerated assessment of investigational therapies
addressing unmet medical need.
The Company’s other late stage drug candidate is CCX140, an
inhibitor of the chemokine receptor known as CCR2, which is
currently being developed for patients with focal segmental
glomerulosclerosis (FSGS), a debilitating kidney disease.
ChemoCentryx’s Kidney Health Alliance with Vifor Pharma provides
Vifor Pharma with exclusive rights to commercialize avacopan and
CCX140 in markets outside of the U.S. and China.
ChemoCentryx also has early stage drug candidates that target
chemoattractant receptors in other Inflammatory and autoimmune
diseases and in cancer.
Forward-Looking Statements
ChemoCentryx cautions that statements included in this press
release that are not a description of historical facts are
forward-looking statements. Words such as "may," "could," "will,"
"would," "should," "expect," "plan," "anticipate," "believe,"
"estimate," "intend," "predict," "seek," "contemplate,"
"potential," "continue" or "project" or the negative of these terms
or other comparable terminology are intended to identify
forward-looking statements. These statements include the Company's
statements whether avacopan (CCX168) will be shown to be safe and
effective in the treatment of C3 glomerulopathy and other rare
diseases and the Company’s statement regarding the timing of
initiating additional clinical trials to further investigate
avacopan in the treatment of C3G. The inclusion of forward-looking
statements should not be regarded as a representation
by ChemoCentryx that any of its plans will be achieved.
Actual results may differ from those set forth in this release due
to the risks and uncertainties inherent in
the ChemoCentryx business and other risks described in
the Company's filings with the Securities and Exchange
Commission ("SEC"). Investors are cautioned not to place undue
reliance on these forward-looking statements, which speak only as
of the date hereof, and ChemoCentryx undertakes no
obligation to revise or update this news release to reflect events
or circumstances after the date hereof. Further information
regarding these and other risks is included under the heading "Risk
Factors" in ChemoCentryx's periodic reports filed with
the SEC, including ChemoCentryx's Annual Report on
Form 10-K filed with the SEC on March 14, 2017 and
its other reports which are available from
the SEC's website (www.sec.gov) and
on ChemoCentryx's website (www.chemocentryx.com) under
the heading "Investors." All forward-looking statements are
qualified in their entirety by this cautionary statement. This
caution is made under the safe harbor provisions of Section 21E of
the Private Securities Litigation Reform Act of 1995.
Contacts:
Susan M. Kanaya
Executive Vice President
Chief Financial and Administrative Officer
investor@chemocentryx.com
Media:
Denise Powell
denise@redhousecomms.com
510.703.9491
Investors:
Steve Klass, Burns McClellan
212.213.0006
sklass@burnsmc.com
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